News

Agios Pharmaceuticals continues to make remarkable strides in developing new treatments for rare blood disorders. The company’s latest quarterly report for Q2 2024 showcases impressive progress for several diseases: ▪️ …

New research suggests Alexion Pharmaceuticals, Inc.‘ immunosuppressive medication, eculizumab, may safely manage hyperhemolysis syndrome (HHS) in pediatric sickle cell disease patients. A Pediatric Blood & Cancer study reported that a …

Emmaus Life Sciences reports compliance with FDA postmarketing safety regulations for Endari, its sickle cell disease treatment. After a 2.5-day inspection under the FDA’s Postmarketing Adverse Drug Experience (PADE) program, …

Novartis has reported encouraging results for its experimental SCD treatment, a “glue degrader” small molecule. In animal studies, the drug effectively increased fetal hemoglobin (HbF) levels, crucial for managing SCD …

Novo Nordisk’s experimental hemophilia A treatment, Mim8, has demonstrated positive outcomes in its Phase 3 FRONTIER-2 trial, effectively controlling bleeding. Among patients with prior preventive treatments, 66% on once-weekly and …

In Phase 3 of ENERGIZE-T, Agios Pharmaceuticals’ drug mitapivat successfully boosted hemoglobin levels in some patients with thalassemia, bringing the company closer to potential approval for the first oral treatment …

Multiple Biopharma giants have announced substantial investments in France under the country’s “Choose France” initiative. Pfizer pledged €500 million over five years to focus on clinical research and trials, particularly …

Following their recent FDA approval and more than a decade of development, biotech firm Bluebird Bio has announced the completion of its first cell collection for their Sickle Cell Disease …

In an exciting announcement from the United Kingdom’s National Health Service (NHS), patients with Sickle Cell Disease can now be prescribed Voxelotor, a Pfizer-developed drug that can significantly improve quality …